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Only 1 percent of liver cells would have to be corrected to successfully treat the disease, said Madeux’s physician and study leader, Dr. Paul Harmatz at the Oakland hospital. “How bulletproof is the technology. We’re just learning,” but safety tests have been very good, said Dr. Carl June, a University of Pennsylvania scientist who has done other gene therapy work but was not involved in this study. One worry is that the virus might provoke an immune system attack. In 1999,18-year-old Jesse Gelsinger died in a gene therapy study from that problem, but the new studies use a different virus that’s proved much safer in other experiments. Another worry is that inserting a new gene might have unforeseen effects on other genes. That happened years ago, when researchers used gene therapy to cure some cases of the immune system disorder called “bubble boy” disease. Doctors say built-in genetic safeguards prevent the therapy from working anywhere but the liver, like a seed that only germinates in certain conditions. This experiment is not connected to other, more controversial work being debated to try to edit genes in human embryos to prevent diseases before birth — changes that would be passed down from generation to generation. Madeux’s treatment was to have happened a week earlier, but a small glitch prevented it. He and his fiancee returned to Arizona, but nearly didn’t make it back to Oakland in time for the second attempt because their Sunday flight was canceled and no others were available until Monday, after the treatment was to take place. Scrambling, they finally got a flight to Monterey, California, and a car service took them just over 100 miles north to Oakland. He had the three-hour infusion, surrounded by half a dozen doctors, nurses and others wearing head-to-toe protective garb to lower the risk of giving him any germs. His doctor, Harmatz, spent the night at the hospital to help ensure his patient stayed well. “I’m nervous and excited,” Madeux said as he prepared to leave the hospital. “I’ve been waiting for this my whole life, something that can potentially cure me. .

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